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International Alliance of ALS/MND Associations

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Expanded Access

Every medication or medical device used today underwent extensive testing to ensure its safety and efficacy. The United States set the standard for clinical trials more than 60 years ago, and the process has not changed much since, except to become more complex and lengthy. It can take a decade or more for a drug candidate to reach the clinic, which for many people is too long to wait.

Fortunately, over the past decade, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have taken steps to expedite certain aspects of clinical trials to more quickly deliver important drugs to people in need. In the US, the expanded access pathway (also called “passionate use”) gives people with life-threatening illness early access to drugs that have not finished clinical testing and, in turn, have not yet been approved by the FDA. The EMA supports expanded access and the programs are operated independently by each member of the European Union.

The Conditions for Compassionate Use

Since compassionate use programs involve giving people drugs that have are not proven safe and effective, the FDA always prefers that people participate in a clinical trial first. The FDA and other regulatory agencies take careful steps to make sure they are not putting patients under undue risk. The FDA only qualifies drugs for expanded access available if:

  • The people who would take it have a immediately life-threatening illness and there are no comparable or effective treatments already available to them
  • These people cannot enroll in a clinical trial, whether it is because no trial is available or the are not eligible for any trials
  • The potential benefits of taking the drug outweigh the potential risks
  • Giving the drug to people will not interfere with an ongoing clinical trial

Expanded access comes in many forms. In some cases, the program is designed to bridge the gap between the end of a clinical trial and marketing approval for a large group of people. In other cases, the pathway is used for a smaller group of people who need a drug that is not actively being developed for clinical use. Finally, a doctor may get permission to give an individual an investigational drug in an emergency setting.

Expanded Access for People with ALS/MND

ALS/MND’s status as a severe, life-threatening disease with no effective treatments might qualify certain experimental ALS/MND drugs for expanded access programs.

 

The Risks Associated with Expanded Access Programs

While the FDA and EMA fully support expanded access programs, they prefer that people enter clinical trials because clinical research offer people more protection if the treatment is unsafe or ineffective. Also, clinical trials are the best way to prove a medication’s safety and efficacy.

It’s important to remember that drugs made available as part of an expanded access program have not been approved for use in the clinic — researchers have not yet proven that it is safe and effective. These drugs make produce unexpected side effects. It is important to discuss the risks with your doctor.

Learn more about expanded access from the FDA here.

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Advocacy

  • Advocacy Toolkit

  • Dad

    Dad

  • Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

    Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

  • Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

    Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

  • Mauril Belanger

    Mauril Belanger

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Art Eggert, USA

    Art Eggert, USA

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

  • Michael Lee, Australia

    Michael Lee, Australia

  • Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

  • Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

    Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

  • 727747090571358167

    727747090571358167

  • Lombana, Spain

    Lombana, Spain

  • Shera Mukherjee, Diagnosed 2013,  Asha Ek Hope Foundation, India

    Shera Mukherjee, Diagnosed 2013, Asha Ek Hope Foundation, India

  • IMG_2658

    IMG_2658

  • Roy

    Roy
    roy

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • Ismail Gokcek, Turkey

    Ismail Gokcek, Turkey
    ismail_gokcek_alsmnd_tr

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • Jon Newsome, USA

    Jon Newsome, USA

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Paul Launer, USA

    Paul Launer, USA

  • Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

    Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

  • Zelina Brito, Diagnosed 2018, Brazil

    Zelina Brito, Diagnosed 2018, Brazil

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Wilfried Leusing

    Wilfried Leusing

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

    Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Bob Simonds and Drew O'Neill , Les Turner ALS Foundation, USA

    Bob Simonds and Drew O’Neill , Les Turner ALS Foundation, USA

  • Debbie Craghill, USA

    Debbie Craghill, USA

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

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